Four IMSA alumni working in the field of orphan disease research came to IMSA to share their findings and stories as panelists in the IMSA Great Minds Program® Alumni Panel on Orphan Diseases. The program was sponsored by IMSA’s Alumni Association (IAA), the student A.X.O.N. Neurology Club, the IMSA Development Office, the IMSA Great Minds Program and the student advocacy group Shine On.
|Dr. Jennifer Ellis Ward '93|
|IMSA Great Minds Program Alumni Panelists with IMSA Principal Dr. Eric McLaren|
|IMSA President Dr. Max McGee|
An orphan disease is a rare disease which has not been "adopted" by the pharmaceutical industry because there are not enough patients to make drug development cost-effective. An orphan disease also can be a disease which is rare in the industrialized world but common and largely ignored in the developing world. These diseases face special challenges in attracting public attention, research and funding. Examples of serious orphan diseases are amyotrophic lateral sclerosis (ALS), cystic fibrosis and Tourette’s syndrome.
To encourage industry to pursue treatments for rare diseases, Congress passed the Orphan Drug Act (ODA) in 1983 to provide financial and regulatory incentives to develop and market therapeutics for diseases that affect less than 200,000 Americans. Since the passage of the ODA in 1983, the FDA has approved more than 350 therapies for orphan diseases. Despite this progress, there are still an estimated 6,000 – 7,000 (or more) orphan diseases in need of effective treatments.
During the presentation, the following alumni panelists shared their perspectives, stories and research findings with students, staff and guests:
Dr. Irfan Qureshi ‘94, an assistant professor in the Department of Neurology and an investigator for Brain Disorders and Neural Regeneration at the Albert Einstein College of Medicine.
Dr. Jennifer Ellis Ward ‘93, a post-doctoral fellow in the Hematology/Oncology Section at the Boston University School of Medicine.
Dr. Michael Wilson ‘94, a neuro-infectious disease fellow at Massachusetts General Hospital and at Boston University’s National Emerging Infectious Diseases Laboratories (NEIDL). NEIDL is the federal government’s newest biosafety level 4 facility, designed to study the most dangerous and deadly infectious agents.
Dr. Meiye Wu ‘94, a staff scientist in the Department of Biotechnology and Bioengineering at Sandia National Laboratory in Livermore, CA.
In addition, David Lockhart ’90, a biostatistician, visited with students throughout the day to share his knowledge. Lockhart is the author of several peer-reviewed articles analyzing data on HIV, AIDS, Alzheimers, cardiac radiology and many other topics.
Dr. Ward, who developed her interest in research as an IMSA student, spoke about her interest in ‘re-purposing old drugs’ for new uses. In addition to being more cost-effective, re-purposing old drugs can also save time in the fight to find cures to orphan diseases.
“There are a lot of challenges in rare disease research … the overwhelmingly biggest one from my perspective is money,” said Dr. Ward. “Drug development and drug discovery … is incredibly expensive. When I first got involved with drug development the saying was ‘it takes 10 years and 10 million dollars to develop a new drug.’ Those numbers are significantly higher now.”
Dr. Irfan Qureshi spoke about epigenetics as “the next frontier” in scientific research. Referencing the Time magazine article, “Why Your DNA Is Not Your Destiny,” Dr Qureshi spoke about how genes and the environment can interact and influence disease acquisition.
“What this means is that for almost every disease, there is an environmental component,” he said. “This is particularly true for those of the nervous system because the nervous system is more influenced by the environment than almost any other system.”
Dr. Qureshi went on to say that epigenetic therapeutic agents can “reprogram neural cells” and that most major drug companies have an epigenetic program.
IMSA President Max McGee applauded IMSA alumni panelists for advancing humanity through their amazing research contributions and reiterated a point to IMSA students in the audience.
“IMSA is educating you for careers that don’t yet exist,” Dr. McGee said. “Twenty years ago, epigenetics didn’t exist.”
To see the alumni presentations, visit http://coolhub.imsa.edu/web/great-minds/.
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